As FDA and stakeholders begin preparations for the reauthorization of the Prescription Drug User Fee Act (PDUFA), Friends of Cancer Research’s new Data Driven-Insight provides a high-level overview of the program’s history, impact, and upcoming priorities. This primer provides essential context as public discussions begin around PDUFA VIII. It also highlights how user fee programs have evolved to support scientific innovation and ensure timely patient access to safe and effective treatments.
What is PDUFA and Why Does It Matter?
The Prescription Drug User Fee Act (PDUFA) provides the U.S. Food and Drug Administration (FDA) with critical supplemental resources to support efficient, predictable, and transparent review timelines and regulatory processes. Since the enactment of PDUFA in 1992, user fees have helped cut New Drug Application (NDA) and Biologics Licensing Application (BLA) priority and standard review times in half, helping get new medicines to patients faster (Figure 1).
Figure 1. Median Review Times for New Drug Applications and Biologics License Applications (1985-2024).[1] PDUFA established performance goals with specific timelines for regulatory reviews, providing a predictable and transparent process that facilitates more efficient regulatory interactions and decision–making.
Expansion of User Fee Programs
Building on the success of PDUFA, user fee programs have also been established to provide supplemental resources for FDA activities for medical products including medical devices (MDUFA, 2002), biosimilars (BsUFA, 2012), and generics (GDUFA, 2012), and over-the-counter monograph drugs (OMUFA, 2020). Over time, the reauthorization of these user fee programs – which occurs every 5 years – has expanded applicability to support key scientific programming related to drug safety surveillance, patient-focused drug development, and other regulatory and scientific modernization efforts (Table 1).
Table 1. History of PDUFA Reauthorization, Review Goals, and Key Initiatives
| PDUFA Cycle | Standard NDA/BLA Review Goal | Priority NDA/BLA Review Goal | Key Initiatives & Changes |
| PDUFA
1993–1997 |
90% in 12 months | 90% in 6 months | Initial authorization – established user fee program and review timelines |
| PDUFA II
1998–2002 |
90% in 12 months; gradually reduced to 10 months by
FY02 |
90% in 6 months | Added goals for efficacy/manufacturing supplements, resubmissions |
| PDUFA III
2002–2007 |
90% in 10 months | 90% in 6 months | Introduced goals for meeting management, IT improvements |
| PDUFA IV (FDAAA) 2008–2012 | 90% in 10 months | 90% in 6 months | Enhanced post-market safety commitments and monitoring, additional meeting types for increased sponsor-FDA collaboration |
| PDUFA V (FDASIA)
2013–2017 |
90% in 10 months | 90% in 6 months | Patient-focused drug development, expanded communication goals,
Set electronic data standards |
| PDUFA VI
(FDARA) 2018–2022 |
90% in 10 months | 90% in 6 months | Real-time review pilots, patient-focused drug development (PFDD) meetings, Model-Informed Drug Development (MIDD) paired meeting program |
| PDUFA VII
2023–2027 |
90% in 10 months | 90% in 6 months | New meeting types (INTERACT, Type D), Split Real Time Application Review (STAR) pilot, rare disease focus (RDEA) |
| PDUFA VIII
2028–2032 |
Reauthorization discussions for the next user fee cycle are beginning in 2025, with PDUFA VIII set to be reauthorized in 2027. | ||
Advancing Innovation through Pilot Programs
PDUFA reauthorizations have included a variety of pilot programs to provide additional opportunities for interaction around innovative scientific methods and trial designs (e.g., CID pilot, RDEA pilot, MIDD paired meeting program) and explore streamlined regulatory approaches (e.g., STAR pilot) (Table 2). These initiatives help advance scientific and regulatory priorities, making FDA a global leader in medical product oversight.
Table 2. Examples of PDUFA-Supported Pilot Projects for Advancing Scientific Innovation
| Program | PDUFA Cycle | Goal | Features/Benefits |
| Split Real Time Application Review (STAR) Pilot Program | PDUFA VII (2023-2027) | Seeks to expedite patient access to novel uses for existing therapies by supporting initiation of review earlier than would otherwise occur and therefore allowing earlier approval for qualified efficacy supplements. |
|
| Rare Disease Endpoint Advancement (RDEA) Pilot Program | PDUFA VII (2023-2027) | Advance rare disease drug development programs by providing a mechanism for sponsors to collaborate with FDA throughout the efficacy endpoint development process.
|
|
| Model Informed Drug Development (MIDD) Paired Meeting Program | Piloted under PDUFA VI (2018-2022)
Continuation under PDUFA VII (2023-2027)
|
Advance and integrate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources in drug development and regulatory review. |
|
| Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot
|
PDUFA VII (2023-2027) | Facilitates CMC readiness for CBER- and CDER-regulated products with accelerated clinical development timelines.
|
|
| Complex Innovative Design (CID) Pilot
|
PDUFA VI (2018-2022) | Advance highly innovative trial designs for which analytically derived properties (e.g., Type I error) may not be feasible, and simulations are necessary to determine trial operating characteristics. |
|
Looking Ahead to PDUFA VIII
Timely reauthorization of PDUFA VIII will ensure FDA continues to have critical resources necessary to uphold its role as a global leader in medical product oversight. Continued investment in regulatory science, innovation, and efficiency will be essential to supporting FDA’s gold-standard approach to protecting and promoting public health.
[1] U.S. FDA. Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals. https://www.fda.gov/drugs/drug-approvals-and-databases/compilation-cder-new-molecular-entity-nme-drug-and-new-biologic-approvals